For the first time, scientists say they have successfully slowed the progression of Huntington’s disease, one of the most devastating inherited brain disorders. The breakthrough gene therapy, delivered through delicate brain surgery, reduced the decline associated with the disease by an average of 75 percent, giving patients the possibility of decades of improved quality of life.
Huntington’s disease is caused by a mutation in the huntingtin gene, which turns a normal protein into a killer of brain cells. Symptoms typically appear in a person’s 30s or 40s and worsen relentlessly, combining traits of dementia, Parkinson’s, and motor neurone disease. The new treatment uses a modified virus to deliver a fragment of DNA deep into the brain, allowing neurons to produce microRNA that shuts down the harmful protein. The surgery lasts up to 18 hours and targets key brain regions that are affected early in the disease.
Trial data from 29 patients, released by biotech company uniQure, showed that three years after treatment, patients experienced dramatically slower progression of symptoms in cognition, movement, and daily living skills. Tests also confirmed fewer brain cells were dying, measured by reduced levels of neurofilaments in spinal fluid. Some trial participants who were expected to lose mobility remain able to walk, while one who had retired early has returned to work.
Researchers at University College London, led by Professors Sarah Tabrizi and Ed Wild, called the results “spectacular” and “breathtaking.” Tabrizi said she never imagined such a profound effect, while Wild described the findings as deeply emotional after years of uncertainty in Huntington’s research. Both emphasized that the therapy could be life-changing for families carrying the gene.
Although the treatment is considered safe, a few patients developed inflammation that required steroids, and the therapy is expected to come with a high price tag. Even so, experts argue that the impact could justify the cost, much like other multimillion-dollar gene therapies currently approved for rare conditions. With regulatory approval targeted for 2026 in the U.S., scientists believe this is just the beginning — opening the door to future trials aimed at preventing Huntington’s symptoms before they ever appear.
